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Development of a Lifespan-Based Novel Composite Person-Reported Outcome Measure Using Data from the CINRG Duchenne Natural History Study

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University of California Davis Sacramento United States

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Development of novel technologies and therapeutic agents to treat Duchenne muscular dystrophy DMD have increased interest by regulatory bodies such as the Food and Drug Administration in the development of clinically-meaningful study endpoints for clinical trials. There is a need for the development of person-reported outcome PRO instruments that target a broad range of developmental and functional ability while effectively evaluating treatment effects in clinical trials. Our proposed project will use quality of life questionnaire data from the first 4-7 years of ongoing Cooperative International Neuromuscular Research Group CINRG Duchenne Natural History Study. Using that data, we will identify questions that show differences between people with different levels of abilities such as those who can walk or just raise a hand to the mouth, or that show changes over one year that might be seen by researchers during drug clinical trials. Those questions will then be combined and built into a computerized adaptive testing CAT system that will produce short, individualized surveys for clinical practice and clinical trial use that are tailored to a patients level of functional ability.

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Technical Report,30 Sep 2014,29 Sep 2019



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Approved For Public Release;

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